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The result was a deeper understanding of complement biology and the ability to apply it to treat diseases in ways no one thought possible. Patient-focused mindset creates medicines that matter As the first to explore the connection between complement biology and rare disease, every phase of the development process — from initial research through clinical trials to delivering new medicines — had to be reimagined. Forging deep connections with patient communities was critical to ensuring unique needs were being addressed as well as establishing a strong foundation for Alexion's research. Alexion's revolutionary 30- year journey has opened new doors for patients and their families and spurred innovation within the rare disease space. The company's research and development advances forward, with potential to address conditions long overlooked or for which a significant need still exists, including several rare conditions, such as lupus nephritis and dermatomyositis. Collaboration is vital to innovation Alexion is surrounded by a growing group of some of the brightest industry researchers and academic community partners who share a commitment to better understanding the role of complement biology in diseases, as well as advancing the science with life-changing therapies to control the dysregulated cascade. The early success Alexion had was in part due to the alliances built between research and academic visionaries that shared a common purpose to find new therapies for patients in need. These alliances have continued to support the incredible potential of complement science, empowering Alexion and its partners to develop a robust research and development engine around complement inhibition with research in more than 15 different indications. Alexion continues to drive this momentum forward, finding new targets, new molecules, and new diseases with the goal to make a difference for patients. With patients as its guiding star every step of the way, Alexion has an opportunity to uncover answers to some of the toughest questions surrounding rare disease and continue to deliver transformative medicines. T he bar for innovation has never been higher. Regardless of the industry, today's challenges require an unparalleled level of ingenuity and a diversity of skillsets and viewpoints that can be achieved only through collaboration. Take the pursuit of treatments for rare disease. According to the National Institutes of Health, 7,000 rare diseases impact approximately 30 million people in the United States alone, yet most rare diseases do not have an approved treatment option. Scientists at Alexion have made it their mission to pioneer new scientific breakthroughs to transform the treatment of rare diseases and devastating conditions. But delivering groundbreaking new medicines takes a cooperative effort and is achievable only when experts from industry and academia work together. Unconventional thinking and perseverance fosters breakthroughs The foundation of Alexion's research began in the early 1990s with a growing body of evidence suggesting a connection between certain diseases and a specific part of the immune system known as the complement system. When operating properly, the complement system plays an integral role in keeping the body healthy and fighting infection. But when the system is thrown out of balance, or dysregulated, it can trigger a series of reactions that attack the body and lead to disease. The scientific community viewed the idea of inhibiting the complement system with skepticism, but Alexion believed in its possibility for patients with few to no treatment options. The potential impact of the science paired with compelling early evidence inspired Alexion to further the research and develop a clinical trial program. Together with leading experts and academics, Alexion strove to validate the clinical utility of complement inhibition. Fueling innovation: What following the science has taught one biotech By Sharon Barr, Ph.D., Senior Vice President, Head of Research and Product Development and Gianluca Pirozzi, M.D., Senior Vice President, Head of Development and Safety, Alexion, AstraZeneca Rare Disease SPONSORED CONTENT Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life- changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology, and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. To learn more about Alexion's work in rare disease, visit Alexion.com.

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